Study of Arimoclomol in Inclusion Body Myositis (IBM)

NCT02753530 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: STUDY00002461R01FD004809
• Study Type: interventional
• Target: 152
• Locations: 2 countries
• Sites: 12

Brief Summary

Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.

Conditions

Inclusion Body Myositis

Keywords

IBM

Outcome Measures

Primary Outcomes (1)

• Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

  Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10-item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.

  Change from Baseline to Month 20

Secondary Outcomes (15)

• Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

  Change from Baseline to Month 12

• Grip Strength

  Change from Baseline to Month 12 and 20

• Modified Timed up and go (mTUG)

  Change from Baseline to Month 12 and Month 20

• Manual Muscle Testing (MMT), Total Score

  Change from Baseline to Month 12 and Month 20

• 6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD)

  Change from Baseline to Month 12 and Month 20

Study Arms (2)

Arimoclomol

EXPERIMENTAL

248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily

Drug: Arimoclomol

Placebo

PLACEBO COMPARATOR

248 mg matching placebo 3 times daily

Other: Placebo

Interventions

Arimoclomol DRUG

2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily during breakfast, early afternoon, and at bedtime

Arimoclomol

Placebo OTHER

2 matched placebo capsules taken 3 times daily during breakfast, early afternoon, and at bedtime

Placebo

Eligibility Criteria

• Age: 45 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Demonstrate being able to arise from a chair without support from another person or device.
• Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, can, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
• Age at onset of weakness \>45 years.
• Body weight of \>= 40 kg.
• Able to give informed consent.

You may not qualify if:

• History of any of the following excludes subject participation in the study: chronic infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer less than five years prior; or other chronic serious medical illnesses.
• Presence of any of the following on routine blood screening: White blood cells (WBC) \<3000; platelets \< 100,000; hematocrit \<30%; blood urea nitrogen (BUN) \>30 mg/dL; creatinine \>1.5 x upper limit of normal; symptomatic liver disease with serum albumin \<3 g/dL.
• History of most recent creatine kinase \>15x the upper limit of normal without any other explanation besides IBM.
• History of non-compliance with other therapies.
• Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
• Coexistence of other disease that would be likely to affect outcome measures
• Drug or alcohol abuse within past three months.
• Participation in a recent drug study in the last 30 days prior to the screening visit or use of a biologic agent less than 6 months prior to the screening visit.
• Women who are lactating or unwilling to use adequate method of birth control who are not surgically sterile. Adequate birth control includes use of intrauterine device, abstinence, or oral contraceptives or a double barrier method, e.g condom plus diaphragm will be necessary for both male and female participants.
• Participants taking \>7.5 mg prednisolone or equivalent or participants on Intravenous Immunoglobulin Therapy (IVIg) or other immunosuppressants within the last 3 months.
• Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment.

Sponsors & Collaborators

• ZevraDenmark: lead
• University of Kansas Medical Center: collaborator
• University College, London: collaborator

Study Sites (12)

Phoenix Neurological Associates

Phoenix, Arizona, 85018, United States

Location

University of California, Irvine

Irvine, California, 92697, United States

Location

University of Colorado School of Medicine

Aurora, Colorado, 80045, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21218, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

The Ohio State University

Columbus, Ohio, 43221, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

University of Virginia

Charlottesville, Virginia, 22908, United States

Location

University College of London

London, WC1N 3BG, United Kingdom

Location

Related Publications (2)

• Salam S, Symonds T, Doll H, Rousell S, Randall J, Lloyd-Price L, Hudgens S, Guldberg C, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM, Machado PM; Members of the Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. Measurement properties of the Inclusion Body Myositis Functional Rating Scale. J Neurol Neurosurg Psychiatry. 2025 Jan 16;96(2):122-131. doi: 10.1136/jnnp-2024-333617.

  PMID: 38960586 DERIVED

• Machado PM, McDermott MP, Blaettler T, Sundgreen C, Amato AA, Ciafaloni E, Freimer M, Gibson SB, Jones SM, Levine TD, Lloyd TE, Mozaffar T, Shaibani AI, Wicklund M, Rosholm A, Carstensen TD, Bonefeld K, Jorgensen AN, Phonekeo K, Heim AJ, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM; Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2023 Oct;22(10):900-911. doi: 10.1016/S1474-4422(23)00275-2.

  PMID: 37739573 DERIVED

MeSH Terms

Conditions

Myositis, Inclusion Body

Interventions

arimoclomol

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Results Point of Contact

• Title: Medical Affairs
• Organization: Zevra Denmark A/S

medicalaffairs@zevra.com

+1-888-289-5607

Study Officials

• Mazen Dimachkie, MD

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

• Michael Hanna, MD

  University College, London

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2016
• First Posted: April 28, 2016
• Study Start: August 16, 2017
• Primary Completion: January 11, 2021
• Study Completion: January 11, 2021
• Last Updated: May 10, 2023
• Results First Posted: May 10, 2023

Record last verified: 2023-04

Locations

GB (1); US (11)